Ann: Neuren receives Rare Pediatric Disease Designation from FDA, page-6

Neuren (NEU) – ASX Announcement 24 February 2025
Neuren receives Rare Pediatric Disease Designation from FDA
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) has received Rare Pediatric Disease
Designation from the US Food and Drug Administration (FDA) for NNZ-2591 in each of Pitt Hopkins
syndrome (PTHS) and Angelman syndrome (AS). Neuren has also previously received Rare Pediatric
Disease Designation from the FDA for NNZ-2591 in Phelan-McDermid syndrome (PMS).
With this designation in place for PMS, PTHS and AS, Neuren may be awarded a priority review voucher
(PRV) if the Rare Pediatric Disease PRV program is reauthorized by the US Congress and NNZ-2591
receives marketing authorisation for any of these indications. The Rare Pediatric Disease PRV program is
designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be
redeemed to receive priority review for a different product or sold to another sponsor. As previously
reported, Neuren’s partner Acadia Pharmaceuticals received a PRV for DAYBUE™ (trofinetide) in Rett
syndrome and sold the PRV for US$150 million.